,,ii  #aag? ,,congress  #b;d ,session   
,s4 #djga  ,,9 ,,! ,,s5ate ,,( ,,!      
,,unit$ ,,/ates  ,april #g1 #bjbb,mr4   
,casey 7= himself & ,mr4 ,scott ( ,s\?  
,c>ol9a7 introduc$ ! foll[+ bill2 :i* 0 
read twice & ref]r$ to ! ,committee on  
,heal?1 ,education1 ,labor1 & ,p5sions  
,a ,,bill  ,to am5d ! ,f$]al ,food1     
,drug1 & ,cosmetic ,act to improve !    
treatm5t ( r>e diseases & conditions1 & 
= o!r purposes4  #a4 ,%ort title ,?is   
,act may be cit$ z ! ,help+ ,exp]ts     
,accel]ate ,r>e ,treatm5ts ,act ( #bjbb4
  #b4 ,improv+ ! treatm5t ( r>e diseases
 & conditions  7a7 ,annual report on    
orphan drug program ,sub*apt] ,b ( *apt]
 ,v ( ! ,f$]al ,food1 ,drug1 & ,cosmetic
 ,act 7#ba ,u4,s4,c4 #cfj;aa et seq47 is
 am5d$ by add+ at ! end ! foll[+ new    
section3  #ebi;,b4 ,annual report on    
orphan drug program  7a7 ,9 g5]al ,n    
lat] ?an #a ye> aft] ! date ( enactm5t (
 ! ,help+ ,exp]ts ,accel]ate ,r>e       
,treatm5ts ,act ( #bjbb1 & n less       
frequ5tly ?an annually !reaft]1 !       

,secret>y %all submit to ,congress a    
report summ>iz+ ! activities ( ! ,food &
 ,drug ,adm9i/ration relat$ to designat+
 drugs "u section #ebf = a r>e disease  
or condition & approv+ su* drugs "u     
section #eje ( ?is ,act or lic5s+ su*   
drugs "u section #cea ( ! ,public ,heal?
 ,s]vice ,act1 includ+-- 7#a7 ! numb] ( 
applications = su* drugs "u section #eje
 ( ?is ,act & section #cea ( ! ,public  
,heal? ,s]vice ,act receiv$ by ! ,food &
 ,drug ,adm9i/ration1 ! numb] ( su*     
applications accept$ = fil+1 ! numb] (  
su* applications reject$ = fil+1 & !    
numb]s ( su* applications p5d+1 approv$1
 & disapprov$ by ! ,food & ,drug        
,adm9i/ration1 array$ by ! review       
division assign$ to ! application2  7#b7
 ! size ( ! affect$ population 9 !      
,unit$ ,/ates ( ea* disease or condition
 address$ by an application describ$ 9  
p>agraph 7#a71 assess$ tak+ into        
consid]ation ! docum5tation requir$ by  
section #caf4bj ( title #ba1 ,code (    
,f$]al ,regulations 7or any successor   

regulation7 & o!r relevant in=mation    
available to ! ,secret>y2 &  7#c7 !     
ext5t ( consultation ) /akehold]s &     
ext]nal exp]ts pursuant to section #efi4
  7b7 ,mann] ( submission ,! in=mation  
requir$ to be report$ "u ?is section may
 be submitt$ z "p ( ano!r report to     
,congress relat$ to ! regulation (      
drugs4  7c7 ,public availability ,!     
,secret>y %all make ea* report "u       
subsection 7a7 available to ! public1   
includ+ by po/+ ! report on ! website ( 
! ,food & ,drug ,adm9i/ration4  4  7b7  
,/udy on ,european ,union safety &      
efficacy reviews ( drugs = r>e diseases 
& conditions  7#a7 ,9 g5]al ,n lat] ?an 
#a ye> aft] ! date ( enactm5t ( ?is     
,act1 ! ,secret>y ( ,heal? & ,human     
,s]vices %all seek to ent] into an      
agreem5t ) ! ,national ,academies (     
,sci5ces1 ,eng9e]+1 & ,m$ic9e 7ref]r$ to
 9 ?is section z ! ,national ,academies7
 to exam9e & report on ,european ,union 
safety & efficacy reviews ( drugs = r>e 
diseases & conditions1 ! use &          

suffici5cy ( exi/+ me*anisms & tools ( !
 ,food & ,drug ,adm9i/ration 9 ensur+ t 
pati5t & physician p]spectives are      
consid]$ ?r\gh\t su* reviews1 &         
opportunities to improve su* reviews 9 !
 ,unit$ ,/ates4  7#b7 ,cont5t ,! report 
develop$ "u p>agraph 7#a7 %all-- 7,a7   
assess & evaluate1 ) respect to drugs = 
r>e diseases & conditions-- 7i7 any     
flexibilities1 au?orities1 or me*anisms 
available 9 ! ,european ,union2  7ii7   
consid]ation & use by ! ,european       
,m$ic9es ,ag5cy ( supplem5tal data      
submitt$ dur+ ! orphan drug application 
review process1 includ+ data associat$ )
 op5 label ext5sion /udies & exp&$      
access programs2 &  7iii7 ea* =mal or   
in=mal process t ! ,food & ,drug        
,adm9i/ration has utiliz$ to ga!r       
ext]nal exp]tise on orphan drug         
applications1 sep>ately consid]+ orphan 
drugs = diseases or conditions t affect 
few] ?an #bj1jjj individuals 9 ! ,unit$ 
,/ates1 comp>$ to orphan drugs = o!r r>e
 diseases or conditions2 &  7,b7 provide

 recomm5dations = *anges to ! processes 
& au?orities ( ! ,food & ,drug          
,adm9i/ration to facilitate developm5t  
(1 & access to1 orphan drugs1 :i* may   
include-- 7i7 new tools or me*anisms to 
improve ef=ts & initiatives ( ! ,food & 
,drug ,adm9i/ration to collect & consid]
 ext]nal exp]tise on orphan drug        
applications1 sep>ately consid]+        
treatm5ts = diseases or conditions t    
affect few] ?an #bj1jjj individuals 9 ! 
,unit$ ,/ates1 includ+ ) respect to     
processes relat$ to application review1 
includ+ /ructur$ b5efit-risk assessm5ts1
 advisory committee delib]ations1 &     
po/approval safety monitor+2 &  7ii7 9 !
 case ( a ne$ = input f ext]nal exp]ts  
:]e !re are limit$ cl9ical & rese>*     
exp]ts available ) respect to a r>e     
disease or condition1 sett+ =?          
alt]native processes to address or      
resolve any conflicts ( int]e/ t w\ld   
o!rwise imp$e inclusion ( input f su*   
ext]nal exp]ts4  7#c7 ,input ,9 conduct+
 ! /udy & develop+ ! report "u p>agraph 

7#b71 ! ,national ,academies %all-- 7,a7
 consid] input f ! ,dep>tm5t ( ,heal? & 
,human ,s]vices1 & any o!r ,f$]al       
ag5cies1 z appropriate2 &  7,b7 consult 
) relevant /akehold]s1 :i* may include  
conduct+ public meet+s & o!r =ms (      
engagem5t1 z appropriate1 ) pati5t      
gr\ps1 heal? c>e provid]s1 m$ical       
pr(essional societies1 public heal?     
exp]ts1 m$ical product manufactur]s1 &  
o!r entities ) relevant exp]i5ce4  7#d7 
,def9itions ,9 ?is subsection-- 7,a7 !  
t]m orphan drug means a drug designat$ z
 a drug = a r>e disease or condition "u 
section #ebf ( ! ,f$]al ,food1 ,drug1 & 
,cosmetic ,act 7#ba ,u4,s4,c4 #cfj;bb72 
&  7,b7 ! t]m r>e disease or condition  
has ! mean+ giv5 su* t]m 9 section #ebf 
( ! ,f$]al ,food1 ,drug1 & ,cosmetic    
,act 7#ba ,u4,s4,c4 #cfj;bb74  7c7      
,review process  7#a7 ,consultation )   
/akehold]s ,section #efi7;a77#a7 ( !    
,f$]al ,food1 ,drug1 & ,cosmetic ,act   
7#ba ,u4,s4,c4 #cfj;bbb-#h7;a77#a77 is  
am5d$-- 7,a7 by /rik+ at a "t & ins]t+  

at any "t2  7,b7 by /rik+ ,consi/5t )   
sections & ins]t+ ! foll[+3  7,a7 ,9    
g5]al ,consi/5t ) sections  2 &  7,c7 by
 add+ at ! end ! foll[+3  7,b7          
,consultation ) pati5ts & pati5t gr\ps  
7i7 ,9 g5]al ,! ,secret>y may1 z        
appropriate1 consult ) pati5ts &        
relevant pati5t gr\ps impact$ by ! r>e  
disease or condition1 toge!r ) at lea/  
"o exp]t includ$ on ! li/ "u p>agraph   
7#b77;,a7 & select$ by su* gr\ps-- 7,i7 
dur+ ! review process ( an application =
 a new drug or biological product = a   
r>e disease or condition or a drug or   
biological product t is g5etically      
t>get$2 &  7,,ii7 z applicable1 dur+    
meet+s betwe5 ! ,food & ,drug           
,adm9i/ration & sponsors prior to !     
submission ( an application = a drug    
describ$ 9 subclause 7,i74  7ii7        
,conflicts ( int]e/ ,= purposes ( clause
 7i71 to be eligible = consultation     
pursuant to clause 7i71 pati5ts &       
relevant pati5t gr\ps may n h any       
f9ancial int]e/ 9 ! applicable drug or  

biological product1 & ext]nal exp]ts    
%all be 9 compliance ) applicable law1  
includ+ section #bjh ( title #ah1 ,unit$
 ,/ates ,code4  7,c7 ,consultation )    
disproportionately affect$ communities  
,to ! ext5t an application = a new drug 
or biological product relates to a r>e  
disease or condition t                  
disproportionately affects communities (
 color or o!r hi/orically und]repres5t$ 
& vuln]able populations1 ! ,secret>y is 
enc\rag$ to consult ) pati5ts ( t       
subpopulation1 or "o or m pati5t gr\ps t
 repres5t t subpopulation4  4  7#b7     
,requir+ appropriate exp]t consultation 
,section #efi7;a77#b7 ( ! ,f$]al ,food1 
,drug1 & ,cosmetic ,act 7#ba ,u4,s4,c4  
#cfj;bbb-#h7;a77#b77 is am5d$-- 7,a7 9  
subp>agraph 7,a71 by /rik+ ! second     
s5t5ce2 &  7,b7 by /rik+ subp>agraph    
7,b7 & ins]t+ ! foll[+3  7,b7           
,consultation ,) respect to any         
application "u section #eje ( ?is ,act  
or section #cea ( ! ,public ,heal?      
,s]vice ,act = a drug designat$ "u      

section #ebf = a r>e disease or         
condition or a drug or biological       
product t is g5etically t>get$1 !       
,secret>y may1 z appropriate1 consult-- 
7i7 ) an exp]t ) respect to ! disease or
 condition ref]5c$ 9 ! application :o   
appe>s on ! li/ describ$ 9 subp>agraph  
7,a72 or  7ii7 if no su* exp]t is       
available1 includ+ because ( conflicts (
 int]e/1 ) an exp]t on ! li/ describ$ 9 
subp>agraph 7,a7 9 ! sci5ce ( small     
population /udies4  7,c7 ,availability  
at meet+s ,9 connection ) ea* drug      
product advisory committee meet+ conc]n+
 a drug or biological product = a r>e   
disease or condition1 ! ,secret>y may1 z
 appropriate-- 7i7 include-- 7,i7 an    
exp]t 9 ! r>e disease or condition2 or  
7,,ii7 if no su* exp]t is available1    
includ+ because ( conflicts ( int]e/1 an
 exp]t 9 ! sci5ce ( small population    
/udies2 &  7ii7 invite at lea/ "o       
disease or condition exp]t id5tifi$ by !
 relevant pati5t gr\ps to p>ticipate z a
 nonvot+ memb] ( ! advisory committee4  

4  7#c7 ,additional topic = consultation
 ,section #efi7;b7 ( ! ,f$]al ,food1    
,drug1 & ,cosmetic ,act 7#ba ,u4,s4,c4  
#cfj;bbb-#h7;b77 is am5d$-- 7,a7 9      
p>agraph 7#f71 by /rik+ 2 & & ins]t+ 22 
7,b7 9 p>agraph 7#g71 by /rik+ ! p]iod &
 ins]t+ 2 &2 &  7,c7 by add+ at ! end ! 
foll[+3  7#h7 ! sci5ce ( small          
population /udies4  4